$2.1 million drug to treat rare genetic disease approved by FDA

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By Linda Carroll and Lauren Dunn

The Food and Drug Administration has approved a drug to treat a rare and fatal genetic disease in children, a life-saving therapy that will cost $2.1 million. The newly approved drug, called Zolgensma, is used to treat children with spinal muscular atrophy, or SMA.

While some doctors argue that this “miracle drug” is too costly, parents of children with SMA may see the new therapy as a “godsend.”

Nicole Almeida, for example, was five months pregnant when doctors told her that the baby she was carrying most likely wouldn’t make it past his first birthday.

“Diagnosis day was and still is one of the worst days of our lives,” Almeida remembers. “It’s like I had the wind knocked out of me.”

Matteo Almeida celebrates his third birthday. Matteo received the experimental drug Zolgensma when he was 27 days old.Courtesy Almeida family

In babies with SMA, nerve cells in the brain stem and spinal cord that control the muscles needed for speaking, walking, breathing and swallowing are destroyed. As the disease progresses, muscles weaken and atrophy, according to the National Institute of Neurological Disorders and Stroke.

Eventually, patients lose the ability to walk, eat or even breathe. That all happens quickly in babies born with the most severe form of the disease—like the Almeidas’ baby, Matteo.

SMA affects approximately 1 in 11,000 babies, and about 1 in every 50 Americans is a genetic carrier, according to SMA Cure, an organization dedicated to finding a cure for the fatal disease.